Affiliation:
1. Department of Biostatistics & Bioinformatics Duke University Durham North Carolina USA
2. Department of Neurological Sciences Rush University Medical Center Chicago Illinois USA
3. Division of Neurology, Department of Medicine, Ottawa Hospital Research Institute, University of Ottawa Brain and Mind Research Institute University of Ottawa Ottawa Ontario Canada
Abstract
AbstractBackgroundRegulatory recommendations favor outcomes combining objective and patient input. The Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS‐UPDRS), the most commonly used scale in Parkinson's disease (PD), includes patient and investigator ratings in distinct parts, but original clinimetric analyses failed to confirm the validity of combining parts by simple summing.ObjectivesThe aim was to develop clinimetrically valid constructs for combining patient‐reported Part 2 and investigator‐rated Part 3 MDS‐UPDRS scores.MethodsUsing 7888 MDS‐UPDRS scores, we assessed construct validity of combined Part 2 and Part 3 items using exploratory factor analysis (EFA) and graded item response theory (IRT) with threshold criteria: comparative fit index ≥0.9 (EFA) and discrimination parameters ≥0.65 (IRT).ResultsThe direct sum of Parts 2 + 3 failed to meet the threshold for a valid outcome of PD severity (comparative fit index, CFI = 0.855). However, a two‐domain construct combining item scores for tremor and non‐tremor domains from Parts 2 and 3 confirmed validity, meeting both EFA and IRT criteria as distinct but correlated indices of disease severity (CFI = 0.923; discrimination mean 2.197 ± 0.480 [tremor] and 1.737 ± 0.344 [non‐tremor] domains).ConclusionsThe sum of Parts 2 + 3 is not clinimetrically sound. However, considering tremor and non‐tremor items of both Parts 2 and 3 as two outcomes results in a valid summary of PD motor severity that leverages simultaneous patient‐ and investigator‐derived measures. This analytic application addresses regulatory prioritizations and retains the well‐validated MDS‐UPDRS items. In future interventional trials, we suggest that tremor and non‐tremor components of PD motor severity from Parts 2 + 3 be monitored and analyzed to accurately detect objective changes that integrate the patient's voice. © 2023 International Parkinson and Movement Disorder Society.
Funder
National Institute on Aging
Subject
Neurology (clinical),Neurology
Cited by
3 articles.
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